WASHINGTON, DC – In an extension of his remarks in the Congressional Record of the House of Representatives, Congressman Gus Bilirakis spoke about vitiligo, the auto-immune disorder that causes depigmentation of the skin, and the efforts of Stella Pavlides, the Founder and President of the American Vitiligo Research Foundation (AVRF).
Rep. Bilirakis noted in his remarks how he learned about vitiligo through Pavlides, one of his constituents in Florida, who is working hard to help raise awareness and funds for research and an eventual cure for this devastating skin disorder.
The AVRF is working with the renowned Dr. David E. Fisher, Chief, Department of Dermatology, Director, Melanoma Program, MGH Cancer Center Director, Cutaneous Biology Research Center at Massachusetts General Hospital, and Edward Wigglesworth Professor of Dermatology at Harvard Medical School. Pavlides shared Dr. Fisher’s most recent letters in support of her efforts.
He writes: “I am writing to share my personal enthusiastic support of the magnificent work being done by Ms. Stella Pavlides who leads the American Vitiligo Research Foundation. I wish to explain the nature of the devastation that this disease causes. Many vitiligo patients are children. Their skin contains unpigmented regions that produce profound alterations in appearance – in unpredictable and frequently widespread fashion. Consequently, vitiligo patients are often subjected to chastisement due to their appearance. Attempts to cover the depigmentation are usually non-permanent, and usually inadequate.
The loss of self-esteem in affected children is heart-wrenching to experience. Some might think that vitiligo has limited seriousness because it is non-lethal. But in fact, vitiligo has been seen to cause suicide as well as permanent devastation to the patient’s interpersonal interactions – from childhood on. It occurs in a significant fraction of the world population, and thus comprises a terribly serious public health problem.
But there is hope. Our understanding of the biology of pigment cells, the immunologic events that cause pigment cell destruction, and new strategies towards restoring pigmentation – are progressing at unprecedented levels. Using the most sophisticated methods of human genome mapping, single-cell based analytic methods, and creative grafting and cell manipulation tools, it has become possible to envision strategies for the future that might have serious impact and hopefully cure this terrible disease. And in the process, these scientific insights are simultaneously informing related conditions – including melanoma and autoimmune diseases.
I end by conveying my deepest support to AVRF and the magnificent work they are doing. Their mission is one of the highest I have known in biomedical research, and I believe that with appropriate resources aimed at supporting vitiligo research, ripe opportunities will be at hand to make a tangible and impactful difference to these children.
Dr. Fisher also noted the novel strategies for the treatment of the skin disorder and also AVRF’s need for funding to support the groundbreaking research: “Vitiligo is a depigmenting condition that arises in approximately 0.5% of the world population. While it is not directly life-threatening, the indirect effects of prominent depigmentation can be devastating. Children affected by vitiligo can suffer severe and permanent loss of self-esteem and adults affected by vitiligo may suffer similar outcomes – often triggering drastic attempts to remove the aberrant appearance, with devastating consequences. Very sadly, suicide is among the tragic results of vitiligo.
Vitiligo is most often caused by ‘auto-immune’ destruction of pigment-producing skin cells (called melanocytes) by an inappropriately activated immune system. Therefore the white patches are the result of diminished numbers of melanocytes, rather than diminished pigment within viable melanocytes. The mainstay of vitiligo treatment has changed very little in over 50 years: use of immunosuppressive therapies such as ultraviolet radiation or topical immunosuppressive drugs, to antagonize the auto-immune process.
However even if the auto-immune process is arrested, ‘re-pigmentation’ is difficult to achieve. The reason is that new melanocytes must become ‘activated’ and must migrate into the depigmented skin, in order to generate new pigment and ameliorate the abnormality. This can occasionally occur, thus offering hope, but it is unpredictable and frequently incomplete and/or non-permanent. Therefore there remains an urgent need for medical strategies to improve patient outcomes.
Despite the long time-period since major advances were made in vitiligo, several key recent discoveries afford opportunities that have never before been available. This proposal seeks to exploit these discoveries. The first of these is the use of “induced pluripotent stem cells” (iPS cells).
The iPS cell technology utilizes gene re-programming to permit normal human skin cells (not from a vitiligo affected skin) to be transformed into cells that can be coaxed to become melanin-producing melanocytes. This technology has been used to generate multiple types of cells (including neurons, blood cells, and heart cells) and won its inventors the Nobel Prize for Medicine in 2012. The use of this technology to generate melanocytes has also been successfully achieved.
The second advance to apply to vitiligo is the discovery of a class of drugs called “SIK-inhibitors” which stimulate melanocytes to produce dark melanin pigment. This discovery was made in 2017 (from our lab at Harvard). While this agent can be helpful to stimulate pigment needed in vitiligo, another key feature is required to successfully treat vitiligo: stimulation of melanocyte “migration” into the unpigmented skin patches.
There is strong suspicion that a pathway known as the Wnt pathway regulates this migration. The Fisher Lab has very recently discovered a class of drugs that may stimulate the Wnt pathway. While it is not yet clear exactly how these drugs stimulate Wnt, it is plausible that they may help – in concert with iPS cells and SIK inhibitors – to produce migratory and pigmented melanocytes, that may provide a profound treatment opportunity for vitiligo patients.
We propose to examine these pathways in detail, if there were funding available from AVRF, with the goal of aiding patients with this devastating disease. No animals will be the subjects of research as part of this work, which will study only human cells within the laboratory.”
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